# N-of-1 and rapid platform trials

Source: https://onco.cc/technologies/n-of-1-platforms/  
OnCo record `n-of-1-platforms` (Technology). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Building a trial around one patient, or letting one trial swap drugs in and out as evidence accumulates.

## Summary

Bespoke antisense drugs have been made for single patients with rare neurological disease under an FDA pathway, and the same logic is being applied to private neoantigen and splice-targeting therapies in cancer. Adaptive platform trials such as I-SPY 2 in breast cancer graduate or drop arms on interim Bayesian analysis, which is the scalable version of the same idea. The obstacles are manufacturing, cost, and how to learn anything generalisable from a single case.

## Fields

- Kind: Technology
- Status: emerging
- Last checked: 2026-09-08
- Tags: frontier; promising
- Principle: Either an intervention is designed for one person's mutation, or a master protocol keeps a shared control arm while arms enter and leave, so each new question costs less.
- Strengths: Reaches patients whose subgroup will never be studied; Platform designs reuse the control arm; Faster answers from fewer patients
- Limitations: Extreme cost per patient for true N-of-1; Interpretation without randomisation is fragile; Regulatory and reimbursement paths unclear

## Sources

- FDA: individualised antisense oligonucleotide guidance (page moved; nearest live section): https://www.fda.gov/drugs/

## Connected records

- fronts: [Drug Discovery Platforms](https://onco.cc/fronts/drug-discovery/)
- technologies: [AI trial matching & clinical decision support](https://onco.cc/technologies/ai-trial-matching/), [Functional (ex vivo) drug testing](https://onco.cc/technologies/functional-drug-testing/), [Personalised neoantigen (mRNA) vaccines](https://onco.cc/technologies/neoantigen-mrna-vaccine/)
- terms: [Basket, umbrella, and platform trials](https://onco.cc/terms/basket-umbrella-platform/), [Master protocol (platform, basket and umbrella trials)](https://onco.cc/terms/master-protocol/), [N-of-1 trial](https://onco.cc/terms/n-of-1-trial/), [Response-adaptive randomisation](https://onco.cc/terms/response-adaptive-randomisation/), [Seamless, adaptive and Bayesian trial designs](https://onco.cc/terms/seamless-adaptive/)
- roadmaps: [Radical oncology: what could change the war by 2035](https://onco.cc/roadmaps/frontier-2035/)
- institutions: [OHSU Knight Cancer Institute](https://onco.cc/institutions/ohsu-knight/)

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